-
1
-
-
0028677475
-
Huntington's disease CAG trinucleotide repeats in pathologically confirmed post-mortem brains
-
Persichetti, F, Srinidhi, J, Kanaley, L, Ge, P, Myers, RH, D'Arrigo, K et al. (1994). Huntington's disease CAG trinucleotide repeats in pathologically confirmed post-mortem brains. Neurobiol Dis 1: 159-166.
-
(1994)
Neurobiol Dis
, vol.1
, pp. 159-166
-
-
Persichetti, F.1
Srinidhi, J.2
Kanaley, L.3
Ge, P.4
Myers, R.H.5
D'Arrigo, K.6
-
2
-
-
47549105506
-
Identification and allele-specific silencing of the mutant huntingtin allele in huntington's disease patient-derived fibroblasts
-
van Bilsen, PH, Jaspers, L, Lombardi, MS, Odekerken, JC, Burright, EN and Kaemmerer, WF (2008). Identification and allele-specific silencing of the mutant huntingtin allele in Huntington's disease patient-derived fibroblasts. Hum Gene Ther 19: 710-719.
-
(2008)
Hum Gene Ther
, vol.19
, pp. 710-719
-
-
Van Bilsen, P.H.1
Jaspers, L.2
Lombardi, M.S.3
Odekerken, J.C.4
Burright, E.N.5
Kaemmerer, W.F.6
-
3
-
-
67349100160
-
Nonallele-specific silencing of mutant and wild-type huntingtin demonstrates therapeutic efficacy in huntington's disease mice
-
Boudreau, RL, McBride, JL, Martins, I, Shen, S, Xing, Y, Carter, BJ et al. (2009). Nonallele-specific silencing of mutant and wild-type huntingtin demonstrates therapeutic efficacy in Huntington's disease mice. Mol Ther 17: 1053-1063.
-
(2009)
Mol Ther
, vol.17
, pp. 1053-1063
-
-
Boudreau, R.L.1
McBride, J.L.2
Martins, I.3
Shen, S.4
Xing, Y.5
Carter, B.J.6
-
4
-
-
65249131740
-
Sustained effects of nonallele-specific huntingtin silencing
-
Drouet, V, Perrin, V, Hassig, R, Dufour, N, Auregan, G, Alves, S et al. (2009). Sustained effects of nonallele-specific Huntingtin silencing. Ann Neurol 65: 276-285.
-
(2009)
Ann Neurol
, vol.65
, pp. 276-285
-
-
Drouet, V.1
Perrin, V.2
Hassig, R.3
Dufour, N.4
Auregan, G.5
Alves, S.6
-
5
-
-
84860192454
-
Six-month partial suppression of huntingtin is well tolerated in the adult rhesus striatum
-
Grondin, R, Kaytor, MD, Ai, Y, Nelson, PT, Thakker, DR, Heisel, J et al. (2012). Six-month partial suppression of Huntingtin is well tolerated in the adult rhesus striatum. Brain 135(Pt 4): 1197-1209.
-
(2012)
Brain
, vol.135
, pp. 1197-1209
-
-
Grondin, R.1
Kaytor, M.D.2
Ai, Y.3
Nelson, P.T.4
Thakker, D.R.5
Heisel, J.6
-
6
-
-
82955199935
-
Preclinical safety of RNAi-mediated HTT suppression in the rhesus macaque as a potential therapy for huntington's disease
-
McBride, JL, Pitzer, MR, Boudreau, RL, Dufour, B, Hobbs, T, Ojeda, SR et al. (2011). Preclinical safety of RNAi-mediated HTT suppression in the rhesus macaque as a potential therapy for Huntington's disease. Mol Ther 19: 2152-2162.
-
(2011)
Mol Ther
, vol.19
, pp. 2152-2162
-
-
McBride, J.L.1
Pitzer, M.R.2
Boudreau, R.L.3
Dufour, B.4
Hobbs, T.5
Ojeda, S.R.6
-
7
-
-
84856228803
-
Widespread suppression of huntingtin with convection-enhanced delivery of siRNA
-
Stiles, DK, Zhang, Z, Ge, P, Nelson, B, Grondin, R, Ai, Y et al. (2012). Widespread suppression of huntingtin with convection-enhanced delivery of siRNA. Exp Neurol 233: 463-471.
-
(2012)
Exp Neurol
, vol.233
, pp. 463-471
-
-
Stiles, D.K.1
Zhang, Z.2
Ge, P.3
Nelson, B.4
Grondin, R.5
Ai, Y.6
-
8
-
-
84883118140
-
Safety and efficacy of RNAi therapy for transthyretin amyloidosis
-
Coelho, T, Adams, D, Silva, A, Lozeron, P, Hawkins, PN, Mant, T et al. (2013). Safety and efficacy of RNAi therapy for transthyretin amyloidosis. N Engl J Med 369: 819-829.
-
(2013)
N Engl J Med
, vol.369
, pp. 819-829
-
-
Coelho, T.1
Adams, D.2
Silva, A.3
Lozeron, P.4
Hawkins, P.N.5
Mant, T.6
-
9
-
-
20244378556
-
RNA interference improves motor and neuropathological abnormalities in a huntington's disease mouse model
-
Harper, SQ, Staber, PD, He, X, Eliason, SL, Martins, IH, Mao, Q et al. (2005). RNA interference improves motor and neuropathological abnormalities in a Huntington's disease mouse model. Proc Natl Acad Sci USA 102: 5820-5825.
-
(2005)
Proc Natl Acad Sci USA
, vol.102
, pp. 5820-5825
-
-
Harper, S.Q.1
Staber, P.D.2
He, X.3
Eliason, S.L.4
Martins, I.H.5
Mao, Q.6
-
10
-
-
84862663712
-
Sustained therapeutic reversal of huntington's disease by transient repression of huntingtin synthesis
-
Kordasiewicz, HB, Stanek, LM, Wancewicz, EV, Mazur, C, McAlonis, MM, Pytel, KA et al. (2012). Sustained therapeutic reversal of Huntington's disease by transient repression of huntingtin synthesis. Neuron 74: 1031-1044.
-
(2012)
Neuron
, vol.74
, pp. 1031-1044
-
-
Kordasiewicz, H.B.1
Stanek, L.M.2
Wancewicz, E.V.3
Mazur, C.4
McAlonis, M.M.5
Pytel, K.A.6
-
11
-
-
33645104092
-
RAAV-mediated shRNA ameliorated neuropathology in huntington disease model mouse
-
Machida, Y, Okada, T, Kurosawa, M, Oyama, F, Ozawa, K and Nukina, N (2006). rAAV-mediated shRNA ameliorated neuropathology in Huntington disease model mouse. Biochem Biophys Res Commun 343: 190-197.
-
(2006)
Biochem Biophys Res Commun
, vol.343
, pp. 190-197
-
-
Machida, Y.1
Okada, T.2
Kurosawa, M.3
Oyama, F.4
Ozawa, K.5
Nukina, N.6
-
12
-
-
25144464388
-
Intrastriatal rAAV-mediated delivery of anti-huntingtin shRNAs induces partial reversal of disease progression in R6/1 huntington's disease transgenic mice
-
Rodriguez-Lebron, E, Denovan-Wright, EM, Nash, K, Lewin, AS and Mandel, RJ (2005). Intrastriatal rAAV-mediated delivery of anti-huntingtin shRNAs induces partial reversal of disease progression in R6/1 Huntington's disease transgenic mice. Mol Ther 12: 618-633.
-
(2005)
Mol Ther
, vol.12
, pp. 618-633
-
-
Rodriguez-Lebron, E.1
Denovan-Wright, E.M.2
Nash, K.3
Lewin, A.S.4
Mandel, R.J.5
-
13
-
-
84862643627
-
"Huntingtin holiday": Progress toward an antisense therapy for Huntington's disease
-
Lu, XH and Yang, XW (2012). "Huntingtin holiday": progress toward an antisense therapy for Huntington's disease. Neuron 74: 964-966.
-
(2012)
Neuron
, vol.74
, pp. 964-966
-
-
Lu, X.H.1
Yang, X.W.2
-
14
-
-
0035710746
-
Analysis of relative gene expression data using realtime quantitative PCR and the 2(-delta delta C(T)) method
-
Livak, KJ and Schmittgen, TD (2001). Analysis of relative gene expression data using realtime quantitative PCR and the 2(-Delta Delta C(T)) Method. Methods 25: 402-408.
-
(2001)
Methods
, vol.25
, pp. 402-408
-
-
Livak, K.J.1
Schmittgen, T.D.2
-
15
-
-
0002196917
-
Comparing individual means in the analysis of variance
-
Tukey, JW (1949). Comparing individual means in the analysis of variance. Biometrics 5: 99-114.
-
(1949)
Biometrics
, vol.5
, pp. 99-114
-
-
Tukey, J.W.1
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